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Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment (APOLLO-IOPD)

Primary Purpose

Glycogen Storage Disease Type II

Status

Completed

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

ALGLUCOSIDASE ALFA (MYOZYME)

About this trial

This is an interventional treatment trial for Glycogen Storage Disease Type II

Eligibility Criteria

0 Months - 12 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion criteria:

Subject's parents or legal guardians must provide written informed consent prior to any study-related procedures.
Documented onset of Pompe disease symptoms up to 12 months of age (corrected for gestation if born before 40 weeks); diagnosis of Pompe disease confirmed by acid alpha-glucosidase enzyme deficiency from any tissue source and acid alpha-glucosidase gene mutations.
Age 0-12 months at enrollment, defined as at the time of providing written informed consent.
Cardiomyopathy (abnormal left ventricular mass indices [LVMIs], measured by echocardiography, abnormal value is defined as ≥65 g/m2 for patients up to 12 months old) confirmed by cardiologist at study site.

Exclusion criteria:

Patient who has previously been treated with acid alpha-glucosidase.
Patient who is participating in another clinical study using any investigational therapy.
Conditions/situations such as:
Clinical signs of cardiac failure with ejection fraction < 40%.
Respiratory insufficiency (oxygen saturation < 90% or carbon dioxide partial pressure > 55 mm Hg [venous] or > 40 mm hydrargyrum [arterial] in room air or any ventilator use).
Patients who are dependent on invasive or non-invasive ventilator support.
Patients with major congenital anomaly or clinically significant intercurrent organic disease unrelated to Pompe disease.
Patients not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sites / Locations

Investigational site number

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Alglucosidase Alfa therapy

Arm Description

Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.

Outcomes

Primary Outcome Measures

Survival

The proportion of patients alive at the end of study

Left Ventricular Mass Index (LVMI)

Change from baseline in LVMI

Secondary Outcome Measures

Invasive ventilation-free survival

Survival free of invasive ventilator use at 52-week treatment

Any ventilation-free survival

Survival free of any ventilator use at 52-week treatment

Growth in body weight and length

Physical growth: Change from baseline at Week 52 with regards to length and weight

Motor development milestones

Number of motor development milestones achieved at Week 52 and change from baseline

GESELL Development Scale

Change from baseline at Week 52 on GESELL Developmental Scale

Cardiac failure

Proportion of patients with signs and/or symptoms of cardiac failure at Week 52

Full Information

NCT ID

NCT03687333

First Posted

September 24, 2018

Last Updated

April 5, 2022

Sponsor

Genzyme, a Sanofi Company

1. Study Identification

Unique Protocol Identification Number

NCT03687333

Brief Title

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Acronym

APOLLO-IOPD

Official Title

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Completed

Study Start Date

December 4, 2018 (Actual)

Primary Completion Date

December 30, 2020 (Actual)

Study Completion Date

December 30, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. Study Description

Brief Summary

Primary Objective: To evaluate effect of 52-week treatment with Alglucosidase Alfa in the extension of survival and improvement of cardiomyopathy measured by Left Ventricular Mass Index in Chinese patients with infantile-onset Pompe Disease. Secondary Objectives: To observe the improvement of physical growth, motor and cognitive development of 52-week treatment with Alglucosidase Alfa in infantile-onset Pompe Disease from the baseline. To observe the efficacy on survival free of invasive ventilation, use of any ventilation support of 52- week treatment with Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease. To evaluate the safety and tolerability of Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.

Detailed Description

Total of 56 weeks in the study period, including an up to 28-day screening period and 52-week treatment period, followed by 30-day post-treatment observation period. After the end of 52-week treatment, patients' guardians could choose to participate in a patient assistance program (PAP) sponsored by Sanofi and launched before first patient out (FPO) or reimbursement from social insurance for continued treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Glycogen Storage Disease Type II

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Alglucosidase Alfa therapy

Arm Type

Experimental

Arm Description

Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.

Intervention Type

Drug

Intervention Name(s)

ALGLUCOSIDASE ALFA (MYOZYME)

Intervention Description

Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

Primary Outcome Measure Information:

Title

Survival

Description

The proportion of patients alive at the end of study

Time Frame

at week 52

Title

Left Ventricular Mass Index (LVMI)

Description

Change from baseline in LVMI

Time Frame

at week 52

Secondary Outcome Measure Information:

Title

Invasive ventilation-free survival

Description

Survival free of invasive ventilator use at 52-week treatment

Time Frame

at week 52

Title

Any ventilation-free survival

Description

Survival free of any ventilator use at 52-week treatment

Time Frame

at week 52

Title

Growth in body weight and length

Description

Physical growth: Change from baseline at Week 52 with regards to length and weight

Time Frame

at week 52

Title

Motor development milestones

Description

Number of motor development milestones achieved at Week 52 and change from baseline

Time Frame

at week 52

Title

GESELL Development Scale

Description

Change from baseline at Week 52 on GESELL Developmental Scale

Time Frame

at week 52

Title

Cardiac failure

Description

Proportion of patients with signs and/or symptoms of cardiac failure at Week 52

Time Frame

at week 52

10. Eligibility

Sex

All

Minimum Age & Unit of Time

0 Months

Maximum Age & Unit of Time

12 Months

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion criteria: Subject's parents or legal guardians must provide written informed consent prior to any study-related procedures. Documented onset of Pompe disease symptoms up to 12 months of age (corrected for gestation if born before 40 weeks); diagnosis of Pompe disease confirmed by acid alpha-glucosidase enzyme deficiency from any tissue source and acid alpha-glucosidase gene mutations. Age 0-12 months at enrollment, defined as at the time of providing written informed consent. Cardiomyopathy (abnormal left ventricular mass indices [LVMIs], measured by echocardiography, abnormal value is defined as ≥65 g/m2 for patients up to 12 months old) confirmed by cardiologist at study site. Exclusion criteria: Patient who has previously been treated with acid alpha-glucosidase. Patient who is participating in another clinical study using any investigational therapy. Conditions/situations such as: Clinical signs of cardiac failure with ejection fraction < 40%. Respiratory insufficiency (oxygen saturation < 90% or carbon dioxide partial pressure > 55 mm Hg [venous] or > 40 mm hydrargyrum [arterial] in room air or any ventilator use). Patients who are dependent on invasive or non-invasive ventilator support. Patients with major congenital anomaly or clinically significant intercurrent organic disease unrelated to Pompe disease. Patients not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Investigational site number

City

Shanghai

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Citations:

PubMed Identifier

35833019

Citation

Zhu D, Zhu J, Qiu W, Wang B, Liu L, Yu X, Ou Z, Shan G, Wang J, Li B, Chen X, Liu C, Li Z, Fu L. A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease. Front Pharmacol. 2022 Jun 27;13:903488. doi: 10.3389/fphar.2022.903488. eCollection 2022.

Results Reference

derived

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Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

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